This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of Cyclosporin A (CsA) in patients with immunologic or histiocytic disorders. The researchers hypothesize that this regimen will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease (GVHD).

Further study details as provided by Masonic Cancer Center, University of Minnesota:

Primary Outcome Measures:

To demonstrate the safety and the ability to establish stable mixed chimerism (>10% donor cells at day 100) using a nonmyeloablative preparative regimen in a phase 2 pilot trial [ Time Frame: Day 100 ] [ Designated as safety issue: Yes ]

Reduced intensity chemotherapy followed by infusion of hematopoietic stem cells

Other Name: hematopoietic stem cell transplant

Drug: Fludarabine, melphalan, ATG or Campath

all drugs are given intravenously (IV). Fludarabine x 5 days and melphalan x 1 day

Other Name: Fludara; Atgam; Campath-1H, Alkeran

Detailed Description:

Prior to transplantation, subjects will receive Melphalan, Fludarabine and Anti-Thymocyte Globulin (ATG) or Campath. These three drugs are being given to subjects to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter.

After stem cell transplantation, subjects will be given Cyclosporin A (CsA) and mycophenolate mofetil (MMF) to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.

Eligibility

Ages Eligible for Study:

up to 35 Years

Genders Eligible for Study:

Both

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Patients with immunodeficiencies or histiocytic disorders 0-35 years of age with an acceptable stem cell donor and disease characteristic defined by the following:

Patients with histocytic disorders (hemophagocytic lymphohistiocytosis of any etiology and refractory Langerhans cell histiocytosis) who do not meet eligibility criteria for a myeloablative transplant procedure

Patients with immunodeficiency disorders in whom residual immune function may not require a fully myeloablative preparative regimen or patient is ineligible for standard myeloablative preparative regimen (any form of severe combined immunodeficiency [SCID], or other immunodeficiency with T cell defect)

Patients with immunodeficiency disorders that have had poor outcome with myeloablative stem cell transplants (including, but not limited to, common variable immunodeficiency [CVID], Wiskott Aldrich Syndrome [WAS] if > 5 years of age, ataxia telangiectasia)

Patients with immunodeficiencies or histocytic disorders that require a second stem cell transplant (SCT) for any reason

Exclusion Criteria:

Karnofsky or Lansky performance score <70

Glomerular filtration rate (GFR)<30% predicted

Cardiac function <50% normal by echocardiogram

Serum creatinine > 2x normal for age/weight

Pregnant or lactating females

Active serious infection that has not had an adequate course of therapy pre-SCT. Any patient with acquired immune deficiency syndrome (AIDS) or AIDS-related complex (ARC) or human immunodeficiency virus (HIV) seropositivity

Contacts and Locations

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Please refer to this study by its ClinicalTrials.gov identifier: NCT00176865